A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD) | MicrobiomeTrials
A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)
RecruitingPHASE1, PHASE2INTERVENTIONAL
Trial Details
Start: 2026-04-15Est. Completion: 2028-09
Enrollment target: 12 participants
Locations
Barrow Neurological Institute, Phoenix, Arizona, United States
University of California, Irvine (UCI), Irvine, California, United States
Stanford Neuroscience Health Center, Palo Alto, California, United States
NYU Langone, New York, New York, United States
Duke University, Durham, North Carolina, United States
Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, United States
University of Pittsburgh Medical Center (UPMC), Pittsburgh, Pennsylvania, United States
University of Texas Southwest Medical Center, Dallas, Texas, United States
Virginia Commonwealth University (VCU), Richmond, Virginia, United States